PMID- 28377044
OWN - NLM
STAT- MEDLINE
DCOM- 20180219
LR  - 20181113
IS  - 1525-0024 (Electronic)
IS  - 1525-0016 (Print)
IS  - 1525-0016 (Linking)
VI  - 25
IP  - 5
DP  - 2017 May 3
TI  - Gene Therapy for beta-Hemoglobinopathies.
PG  - 1142-1154
LID - S1525-0016(17)30123-5 [pii]
LID - 10.1016/j.ymthe.2017.03.024 [doi]
AB  - beta-Thalassemia and sickle cell disease (SCD) are the world's two most widely 
      disseminated hereditary hemoglobinopathies. beta-Thalassemia originated in the 
      Mediterranean, Middle Eastern, and Asian regions, and SCD originated in central 
      Africa. However, subsequent population migration means that these two diseases 
      are now global and thus constitute a growing health problem in many countries. 
      Despite remarkable improvements in medical care for patients with 
      beta-hemoglobinopathies, there is still only one definitive treatment option: 
      allogeneic hematopoietic stem cell (HSC) transplantation. The development of gene 
      therapy for beta-hemoglobinopathies has been justified by (1) the limited 
      availability of human leukocyte antigen (HLA)-identical donors, (2) the narrow 
      window of application of HSC transplantation to the youngest patients, and (3) 
      recent advances in HSC-based gene therapy. The huge ongoing efforts in 
      translational medicine and the high number of related publications show that gene 
      therapy has the potential to become the treatment of choice for patients who lack 
      either an HLA genoidentical sibling or an alternative, medically acceptable 
      donor. In this dynamic scientific context, we first summarize the main steps 
      toward clinical translation of this therapeutic approach and then discuss novel 
      lentiviral- and genome editing-based treatment strategies for 
      beta-hemoglobinopathies.
CI  - Copyright (c) 2017 The American Society of Gene and Cell Therapy. Published by 
      Elsevier Inc. All rights reserved.
FAU - Cavazzana, Marina
AU  - Cavazzana M
AD  - Biotherapy Department, Necker Children's Hospital, Assistance Publique-Hopitaux 
      de Paris, 75015 Paris, France; Biotherapy Clinical Investigation Center, 
      Assistance Publique-Hopitaux de Paris, INSERM, Groupe Hospitalier Universitaire 
      Ouest, 75015 Paris, France; INSERM UMR 1163, Laboratory of Human 
      Lymphohematopoiesis, 75015 Paris, France; Paris Descartes, Sorbonne Paris Cite 
      University, Imagine Institute, 75015 Paris, France. Electronic address: 
      m.cavazzana@aphp.fr.
FAU - Antoniani, Chiara
AU  - Antoniani C
AD  - Paris Descartes, Sorbonne Paris Cite University, Imagine Institute, 75015 Paris, 
      France; INSERM UMR 1163, Laboratory of Chromatin and Gene Regulation, 75015 
      Paris, France.
FAU - Miccio, Annarita
AU  - Miccio A
AD  - Paris Descartes, Sorbonne Paris Cite University, Imagine Institute, 75015 Paris, 
      France; INSERM UMR 1163, Laboratory of Chromatin and Gene Regulation, 75015 
      Paris, France. Electronic address: annarita.miccio@institutimagine.org.
LA  - eng
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
PT  - Review
DEP - 20170401
PL  - United States
TA  - Mol Ther
JT  - Molecular therapy : the journal of the American Society of Gene Therapy
JID - 100890581
RN  - 0 (HLA Antigens)
RN  - 0 (beta-Globins)
SB  - IM
MH  - Anemia, Sickle Cell/genetics/metabolism/pathology/*therapy
MH  - Gammaretrovirus/genetics/immunology
MH  - Gene Editing/*methods
MH  - Gene Expression
MH  - Genetic Therapy/*methods
MH  - Genetic Vectors/chemistry/immunology
MH  - HLA Antigens
MH  - *Hematopoietic Stem Cell Transplantation
MH  - Hematopoietic Stem Cells/cytology/metabolism
MH  - Humans
MH  - Lentivirus/genetics/immunology
MH  - Mutation
MH  - Tissue Donors
MH  - Transplantation, Homologous
MH  - beta-Globins/*genetics/metabolism
MH  - beta-Thalassemia/genetics/metabolism/pathology/*therapy
PMC - PMC5417842
OTO - NOTNLM
OT  - gene therapy
OT  - hematopoietic stem cell
OT  - hemoglobinopathies
OT  - sickle cell disease
OT  - thalassemias
EDAT- 2017/04/06 06:00
MHDA- 2018/02/20 06:00
PMCR- 2018/05/03
CRDT- 2017/04/06 06:00
PHST- 2017/03/14 00:00 [received]
PHST- 2017/03/15 00:00 [revised]
PHST- 2017/03/15 00:00 [accepted]
PHST- 2017/04/06 06:00 [pubmed]
PHST- 2018/02/20 06:00 [medline]
PHST- 2017/04/06 06:00 [entrez]
PHST- 2018/05/03 00:00 [pmc-release]
AID - S1525-0016(17)30123-5 [pii]
AID - 10.1016/j.ymthe.2017.03.024 [doi]
PST - ppublish
SO  - Mol Ther. 2017 May 3;25(5):1142-1154. doi: 10.1016/j.ymthe.2017.03.024. Epub 2017 
      Apr 1.